Acetylsalicylic acid suppression of the PI3K pathway as a novel medical therapy for head and neck lymphatic malformations

dc.contributor.authorBonilla-Velez, Juliana
dc.contributor.authorWhitlock, Kathryn B.
dc.contributor.authorGanti, Sheila
dc.contributor.authorZenner, Kaitlyn
dc.contributor.authorCheng, Chi Vicky
dc.contributor.authorJensen, Dana M.
dc.contributor.authorPham, Minh-Hang M.
dc.contributor.authorMitchell, Ryan M.
dc.contributor.authorDobyns, William
dc.contributor.authorBly, Randall A.
dc.contributor.authorBennett, James T.
dc.contributor.authorDahl, John P.
dc.contributor.authorPerkins, Jonathan A.
dc.contributor.departmentOtolaryngology -- Head and Neck Surgery, School of Medicine
dc.date.accessioned2024-04-05T09:56:17Z
dc.date.available2024-04-05T09:56:17Z
dc.date.issued2021
dc.description.abstractObjectives: Head and neck lymphatic malformations (HNLM) are caused by gain-of-function somatic mutations in PIK3CA. Acetylsalicylic acid (ASA/aspirin) is thought to limit growth in PIK3CA-mutated neoplasms through PI3K pathway suppression. We sought to determine if ASA could be beneficial for HNLM. Methods: Retrospective case series of patients (0-18 years) offered ASA (3-5 mg/kg/day) for HNLM treatment (2010-2018). Clinical and treatment characteristics, patient-reported symptom improvement, medication tolerance, compliance, and complications were recorded. Treatment response was determined by change in patient/caregiver-reported symptoms, or HNLM size [complete (resolved), partial (decreased), or stable]. Results: Fifty-three patients were offered ASA, 23 (43%) accepted (median age 10 years, IQR 6-14). Compared to patients who declined, patients receiving ASA were more likely to have extensive malformations: ex-utero intrapartum treatment procedure, bilateral malformations, oral cavity location, ≥2 invasive treatments, or tracheotomy (p < 0.05). All patients with tissue available had PIK3CA mutations (13/23). Treatment indications included oral pain/blebs (12, 52%), recurrent pain/swelling (6, 26%), or sudden/persistent swelling (5, 22%). Treatment plan was commonly one 81 mg tablet daily (19, 83%) for 3-12 months (8, 42%). Therapeutic adherence was reported by 18 patients (78%). Symptoms improved in 18 patients [78%; decreased pain (9, 39%) and swelling (8, 35%)]. Treatment resulted in partial (14, 61%) or complete response (4, 17%). Three patients developed oral bleb bleeding, which resolved with medication discontinuation. Conclusion: ASA seems to be a well-tolerated, low-risk medication for HNLM treatment. This pilot study suggests that it often improves symptoms and reduces HNLM size. Further prospective, randomized studies are warranted to comprehensively assess indications, safety, and efficacy.
dc.eprint.versionAuthor's manuscript
dc.identifier.citationBonilla-Velez J, Whitlock KB, Ganti S, et al. Acetylsalicylic acid suppression of the PI3K pathway as a novel medical therapy for head and neck lymphatic malformations. Int J Pediatr Otorhinolaryngol. 2021;151:110869. doi:10.1016/j.ijporl.2021.110869
dc.identifier.urihttps://hdl.handle.net/1805/39804
dc.language.isoen_US
dc.publisherElsevier
dc.relation.isversionof10.1016/j.ijporl.2021.110869
dc.relation.journalInternational Journal of Pediatric Otorhinolaryngology
dc.rightsPublisher Policy
dc.sourcePMC
dc.subjectLymphatic malformation
dc.subjectLymphatic abnormalities
dc.subjectAcetylsalicylic acid
dc.subjectPI3K
dc.subjectTreatment
dc.titleAcetylsalicylic acid suppression of the PI3K pathway as a novel medical therapy for head and neck lymphatic malformations
dc.typeArticle
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